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Babies with SMA able to swallow with Evrysdi® treatment
All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
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Regenerative Medicine
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Manufacturing, Packaging & Logistics In-Depth Focus 2021
Features in this in-depth focus highlight some of the important developments in the cell and gene therapy market, as well as the key manufacturing strategies to consider when taking them from concept to commercialisation.
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Dissolvable microneedle patch may be the answer to baldness
A newly developed investigational microneedle patch caused mice to regrow hair faster than minoxidil, a leading alopecia treatment.
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Parkinson’s disease market sales to reach $11.5 billion in 2029
The Parkinson’s disease market is set to triple in sales by 2029 driven by novel biologics and delivery systems, reports GlobalData.
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Ritlecitinib improves scalp hair regrowth in alopecia trial
Ritlecitinib was shown to reduce scalp hair loss to less than or equal to 20 percent after six months in Pfizer’s Phase IIb/III trial.
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Saphnelo approved to treat systemic lupus erythematosus
AstraZeneca’s Saphnelo (anifrolumab-fnia) has been approved in the US for patients with moderate to severe systemic lupus erythematosus.
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Roche’s Evrysdi significantly improves lives of babies with SMA
The Phase II study of Evrysdi showed significant improvements in survival and motor milestones in babies with type 1 spinal muscular atrophy (SMA).
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Alzheimer’s treatment lowers tau by nearly 50 percent in Phase I trials
Biogen and Ionis announced positive topline data on investigational Alzheimer’s treatment, indicating 50 percent tau protein reduction.
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EMA’s human medicine committee (CHMP) meeting highlights, July 2021
The Committee for Medicinal Products for Human Use (CHMP) recommended two medicines for approval and provided a negative opinion on Nouryant.
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Lilly to acquire biotech company Protomer Technologies
Eli Lilly and Company has announced the acquisition of California-based biotech company, Protomer Technologies.
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International Society for Stem Cell Research (ISSCR) releases new guidelines
The ISSCR has released updated guidelines for stem cell research and clinical translation of stem cell-based therapies.
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Stem cell therapy shows promise in COVID-19 respiratory distress
JadiCell™ was shown to significantly improve survival in COVID-19 patients with Acute Respiratory Distress Syndrome (ARDS) and also reduce lung scarring associated with long-COVID-19.
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Curative cell and gene therapies and healthcare system disruption
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
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Stem cell transplantation may be able to prevent major cause of death in preterm infants
Transplanting stem cells directly into the trachea of preterm infants with bronchopulmonary dysplasia was found to be safe and of some benefit in the most vulnerable group.
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How has COVID-19 impacted the development of cell and gene therapies?
European Pharmaceutical Review’s Hannah Balfour explores the findings of the Cell and Gene Therapy (CGT) Catapult’s 2020 advanced therapy medicinal product trials report and discusses how the European and global CGT markets are expected to perform in coming years.
